PhD Scientific Days 2023

Budapest, 22-23 June 2023

Clinical Medicine V.

Real-life Experience of Lanadelumab Prophylaxis After Six Months in Hungarian Patients with Hereditary Angioedema due to C1-inhibitor-deficiency

Hanga Réka Horváth1, Beáta Visy1,2, Henriette Farkas1
1 Hungarian Angioedema Center of Reference and Excellence, Department of Haematology and Internal Medicine, Semmelweis University, Budapest, Hungary
2 Heim Pál National Institute of Pediatrics, Budapest, Hungary

Text of the abstract

Introduction: Hereditary angioedema (HAE) is a rare disease with autosomal dominant inheritance. HAE is characterised by recurrent submucous and/or subcutaneous swellings (HAE attacks) that can be life-threatening and can cause severe psychosocial stress for the patients. In its most common form, the disease is caused by the deficiency of the C1-inhibitor protein. The formation of the angioedema is connected to the over-activation of the plasma kinin-kallikrein system that leads to bradykinin formation. One of the modern, targeted long-term prophylactic treatment options is lanadelumab, a monoclonal antibody against plasma kallikrein, that has to be administered subcutaneously every second week. In 2022 eight Hungarian patients got the chance to be treated with this expensive medication.
Aims: The aim of our study was to evaluate the efficacy and safety of the lanadelumab therapy based on the first six months of treatment.
Methods: Blood and urine tests were taken from our patients before initiation of treatment and at 1, 3 and 6 months of treatment. Each patient has filled out two types of questionnaires regarding their quality of life right before the first dose and every month since then. We examined the occurrence of HAE attacks and the duration of longest attack-free periods in the six months before and the six months since the initiation of lanadelumab. The potential side-effects were also monitored.
Results: Eight patients (4 women and 4 men, median age 39.5 years (min.-max. 16.9-59)) were observed. One patient (who has bipolar disorder) had to stop the treatment after four months because they felt no improvement. The remaining seven patients expressed significant decrease in the number of HAE attacks after the initiation of treatment and a significant elongation of the longest attack-free period (p<0.02 for both). After six months of treatment, the control level of the disease improved significantly, and a significant improvement could be observed in the quality of life of the patients (p<0.02 for both). There was no notable change in the complement parameters during the treatment period. No local or systemic side effects were observed.
Conclusion: Long-term prophylaxis with subcutaneous lanadelumab is a safe and effective way to decrease the number of HAE attacks and improve patients’ quality of life.
Funding: EFOP-3.6.3_VEKOP-16-2017-00009, NKFI 124557